Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting – Business Wire

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: Treatment Presenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles Session Date: Friday, October 16, 2020 Session Time: 10:45 a.m. 12:01 p.m. CEST Lecture Time: 11:45 a.m. CEST Location: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial Initiation Presenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

Huge Investment in Induced Pluripotent Stem Cells Market Expected to Witness the Highest Growth 2026 | Fujifilm Holding Corporation (CDI) Ncardia,…

Induced Pluripotent Stem Cells Market has been growing exponentially over time and has shown great potential in the near future. The growth of Induced Pluripotent Stem Cells Market is expected to see an amazing uproar as the market becomes increasingly popular. The report focuses on the key growth contributors of the market to help the clients better understand the current scenario of the market all while considering the history as well as the forecast of the Induced Pluripotent Stem Cells Market. Essential growth factors have been discussed in the following report.

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Induced Pluripotent Stem Cells Market Type Coverage:

Human iPSCs Mouse iPSCs

Induced Pluripotent Stem Cells Market Application Coverage:

Academic Research Drug Development and Discovery Toxicity Screening Regenerative Medicine

Market Segment by Regions and Nations included:

North America (United States, Canada, Mexico)

Asia-Pacific (China, Japan, Korea, India, Southeast Asia)

South America (Brazil, Argentina, Colombia, etc.)

Europe, Middle East and Africa (Germany, France, UK, Russia and Italy, Saudi Arabia, UAE, Egypt, Nigeria, South Africa)

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Huge Investment in Induced Pluripotent Stem Cells Market Expected to Witness the Highest Growth 2026 | Fujifilm Holding Corporation (CDI) Ncardia,...

Stem Cell-Derived Cells Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2019 – 2029 – Stock Market Funda

In this report, the global Stem Cell-Derived Cells market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2025, growing at a CAGR of XX% during the period 2019 to 2025.

Persistence Market Research recently published a market study that sheds light on the growth prospects of the global Stem Cell-Derived Cells market during the forecast period (20XX-20XX). In addition, the report also includes a detailed analysis of the impact of the novel COVID-19 pandemic on the future prospects of the Stem Cell-Derived Cells market. The report provides a thorough evaluation of the latest trends, market drivers, opportunities, and challenges within the global Stem Cell-Derived Cells market to assist our clients arrive at beneficial business decisions.

The Stem Cell-Derived Cells market report firstly introduced the basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the Stem Cell-Derived Cells market report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

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Resourceful insights enclosed in the report:

The major players profiled in this Stem Cell-Derived Cells market report include:

key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type

Segmentation by End User

The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on:

Regional analysis includes

Report Highlights:

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Stem Cell-Derived Cells Market Forecasted To Surpass The Value Of US$ XX Mn/Bn By 2019 - 2029 - Stock Market Funda

What You Need to Know About Prop 14, The Stem Cell Research Bond (Transcript) – KQED

If reading through the statewide ballot propositions has made your head spin, you are in the right place! From Oct. 1 - 16, Bay Curious is exploring the 12 statewide ballot propositions in our Prop Fest series. This episode tackles Prop 14, the stem cell research bond.

Olivia Allen-Price [00:01:55] OK, so what exactly does this bond fund?

Danielle Venton [00:01:59] This would fund $5.5 billion in stem cell research and treatments in California. Some of the diseases that stem cell research is seeking to cure or treat include cancer, Alzheimer's disease, diabetes, spinal cord injuries, blindness, and even COVID-19. I spoke recently with a guy named Jake Javier. He supports this bond initiative because he knows firsthand how life changing stem cell research can be.

Jake Javier [00:02:25] I am in my last year at Cal Poly.

Danielle Venton [00:02:28] So, Jake grew up locally in Danville and was just graduating high school when he suffered a life altering injury.

Jake Javier [00:02:35] On the last day of high school, I drove in to a pool and hit my head on the bottom and broke my neck and was immediately paralyzed.

Danielle Venton [00:02:47] He says his injury was complete, with very little hope of recovery. But a doctor at Stanford reached out to Jake and his family and said, you can be part of this clinical trial where we, with a one time surgery, will inject stem cells into the damaged area and you may possibly see some benefits.

Danielle Venton [00:03:07] Now, Jake is still injured.

Jake Javier [00:03:09] I'm a quadriplegic. I use a wheelchair.

Danielle Venton [00:03:11] But he says after the surgery, he noticed more movement in his arms, in his hands.

Jake Javier [00:03:17] So, I mean, with my injury, I'm at a level where I would normally not have any function at all in my hands and very, very little function like in my triceps and things like that. Muscles that are really important for functionality and, you know, being able to get through day to day activities that could help me push myself around more, help me transfer in and out of my chair independently. And then also, I notice, you know, I got some some finger movement. It doesn't seem like much, but even that little movement has helped me so much with picking things up and things like that. So it was really, I was really blessed to see that happen.

Danielle Venton [00:03:51] So he doesn't know how much of his recovery is due to the stem cells. How much is natural, or how much is due to physical therapy. But today he's able to live independently, to go to college and he wants to pursue a career in medicine. And he is a big believer in stem cell research, regenerative medicine, and is really hoping that California voters will support this proposition.

Olivia Allen-Price [00:04:20] Now, what exactly are stem cells and how do they work, I guess?

Danielle Venton [00:04:25] Yeah, stem cells are types of cells that can be turned into any type of specialized cell. Scientists have known about them since the eighteen hundreds, but it wasn't until the late 90s that researchers developed a method to derive them from human embryos and grow them in a laboratory. And then people really began to get excited about their potential for medicine. Now these cells came from unused embryos created for in vitro fertilization, and they were donated with informed consent. But many anti-abortion groups felt that using the cells were tantamount to taking a human life. So in 2001, then President George W. Bush banned federal funding for any research using newly created stem cell lines.

Olivia Allen-Price [00:05:09] OK. And how does that get us now to bonds in California?

Danielle Venton [00:05:13] Well, Californians wanted to circumvent these federal restrictions, and in 2004 voted for a bond that gave the state $3 billion to create a research agency called the California Institute of Regenerative Medicine, or CIRM. There was a lot of public support for it. And it just felt like these wonderful cures could be right around the corner. Celebrities like Michael J. Fox appeared in TV commercials.

Michael J. Fox TV commercial [00:05:36] My most important role lately is as an advocate for patients, and for finding new cures for diseases. That's why I'm asking you to vote yes on Proposition 71, Stem Cell Research Initiative.

Danielle Venton [00:05:48] And the money for that research, that $3 billion, has now run out. And to continue their work, the stem cell advocacy group, Americans for Cures, is asking voters for more money.

Olivia Allen-Price [00:06:00] So we're basically voting on whether we want to refill the stem cell research piggy bank here.

Danielle Venton [00:06:05] Yeah, exactly. Some question if the state can afford this at this time when budgets are going to be so tight. Others have been disappointed by the slow pace of cures coming out of the field. Now, there are people who credit this research, such as Jake, with improving or restoring their health or the health of their loved ones. Or maybe they hope that one day it will, and they would balk at the idea that this is not worthy research. They point to achievements that the agency has funded. That includes effectively a cure for bubble baby disease. This is when someone is born without a functioning immune system. That mutation can now be corrected with genetically modified stem cells. And recently, just within the last year or so, the FDA approved two new treatments for blood cancer, developed with CIRM support. These achievements are what the agency points to when they're criticized for not having accomplished more. And they say the process of scientific discovery is long and unpredictable.

Olivia Allen-Price [00:07:04] Now, wasn't that Bush-era ban on stem cell research that you were talking about earlier wasn't that overturned?

Danielle Venton [00:07:11] Yes, that was overturned by President Obama. However, there are current members of Congress who are lobbying President Trump to ban the research again. And if that happens, then California would be the only major player in stemcell research once again in the United States.

Olivia Allen-Price [00:07:30] All right, so who is supporting Prop 14?

Danielle Venton [00:07:32] Governor Gavin Newsom, for one. Many patient advocacy organizations and medical and research institutions, including the California Board of Regents. These people don't want to see the pace of this research slow. They want it to accelerate. The political action committee supporting this proposition is reporting more than six million dollars in contributions.

Olivia Allen-Price [00:07:53] All right. And what about the opposition? Who's against it?

Danielle Venton [00:07:55] Well, so far, there's no organized, funded opposition. There have been several newspaper editorials coming out against it, including locally, the Mercury News and the Santa Rosa Press Democrat. They basically say state bonds aren't the way to fund research and the situation isn't like it was in 2004 and that the institute should now seek other sources of funding and move forward as a nonprofit.

Olivia Allen-Price [00:08:19] All right, Danielle. Well, thanks, as always for your help.

Danielle Venton [00:08:21] My pleasure. Thanks.

Olivia Allen-Price [00:08:28] In a nutshell, a vote yes on Proposition 14 says you think Californians should give $5.5 billion to the state's stem cell research institute. That money will be raised by selling bonds, which the state would pay back, with interest, out of the general fund over the next 30 years. A vote no means you think we shouldn't spend public money on this research.

Olivia Allen-Price [00:08:54] That's it on Proposition 14. We'll be back tomorrow with an episode on Prop 15. And oh, it is a doozy. Commercial property tax! A partial rollback of one of California's most controversial propositions! It's going to be fire. In the meantime, you can find more of KQED election coverage at KQED.org/elections. Two reminders on the way out: October 19th is the last day to register to vote and mail in ballots must be postmarked on or before November 3rd.

Olivia Allen-Price [00:09:28] Bay Curious is made in San Francisco at member supported KQED. I'm Olivia Allen-Price. See you tomorrow.

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What You Need to Know About Prop 14, The Stem Cell Research Bond (Transcript) - KQED

SMART researchers receive Intra-CREATE grant for personalized medicine and cell therapy – MIT News

Researchers from Critical Analytics for Manufacturing Personalized-Medicine (CAMP), an interdisciplinary research group at Singapore-MIT Alliance for Research and Technology (SMART), MITs research enterprise in Singapore, have been awarded Intra-CREATE grants from the National Research Foundation (NRF) Singapore to help support research on retinal biometrics for glaucoma progression and neural cell implantation therapy for spinal cord injuries. The grants are part of the NRFs initiative to bring together researchers from Campus for Research Excellence And Technological Enterprise (CREATE) partner institutions, in order to achieve greater impact from collaborative research efforts.

SMART CAMP was formed in 2019 to focus on ways to produce living cells as medicine delivered to humans to treat a range of illnesses and medical conditions, including tissue degenerative diseases, cancer, and autoimmune disorders.

Singapores well-established biopharmaceutical ecosystem brings with it a thriving research ecosystem that is supported by skilled talents and strong manufacturing capabilities. We are excited to collaborate with our partners in Singapore, bringing together an interdisciplinary group of experts from MIT and Singapore, for new research areas at SMART. In addition to our existing research on our three flagship projects, we hope to develop breakthroughs in manufacturing other cell therapy platforms that will enable better medical treatments and outcomes for society, says Krystyn Van Vliet, co-lead principal investigator at SMART CAMP, professor of materials science and engineering, and associate provost at MIT.

Understanding glaucoma progression for better-targeted treatments

Hosted by SMART CAMP, the first research project, Retinal Analytics via Machine learning aiding Physics (RAMP), brings together an interdisciplinary group of ophthalmologists, data scientists, and optical scientists from SMART, Singapore Eye Research Institute (SERI), Agency for Science, Technology and Research (A*STAR), Duke-NUS Medical School, MIT, and National University of Singapore (NUS). The team will seek to establish first principles-founded and statistically confident models of glaucoma progression in patients. Through retinal biomechanics, the models will enable rapid and reliable forecast of the rate and trajectory of glaucoma progression, leading to better-targeted treatments.

Glaucoma, an eye condition often caused by stress-induced damage over time at the optic nerve head, accounts for 5.1 million of the estimated 38 million blind in the world and 40 percent of blindness in Singapore. Currently, health practitioners face challenges forecasting glaucoma progression and its treatment strategies due to the lack of research and technology that accurately establish the relationship between its properties, such as the elasticity of the retina and optic nerve heads, blood flow, intraocular pressure and, ultimately, damage to the optic nerve head.

The research is co-led by George Barbastathis, principal investigator at SMART CAMP and professor of mechanical engineering at MIT, and Aung Tin, executive director at SERI and professor at the Department of Ophthalmology at NUS. The team includes CAMP principal investigators Nicholas Fang, also a professor of mechanical engineering at MIT; Lisa Tucker-Kellogg, assistant professor with the Cancer and Stem Biology program at Duke-NUS; and Hanry Yu, professor of physiology with the Yong Loo Lin School of Medicine, NUS and CAMPs co-lead principal investigator.

We look forward to leveraging the ideas fostered in SMART CAMP to build data analytics and optical imaging capabilities for this pressing medical challenge of glaucoma prediction, says Barbastathis.

Cell transplantation to treat irreparable spinal cord injury

Engineering Scaffold-Mediated Neural Cell Therapy for Spinal Cord Injury Treatment (ScaNCellS), the second research project, gathers an interdisciplinary group of engineers, cell biologists, and clinician scientists from SMART, Nanyang Technological University (NTU), NUS, IMCB A*STAR, A*STAR, French National Centre for Scientific Research (CNRS), the University of Cambridge, and MIT. The team will seek to design a combined scaffold and neural cell implantation therapy for spinal cord injury treatment that is safe, efficacious, and reproducible, paving the way forward for similar neural cell therapies for other neurological disorders. The project, an intersection of engineering and health, will achieve its goals through an enhanced biological understanding of the regeneration process of nerve tissue and optimized engineering methods to prepare cells and biomaterials for treatment.

Spinal cord injury (SCI), affecting between 250,000 and 500,000 people yearly, is expected to incur higher societal costs as compared to other common conditions such as dementia, multiple sclerosis, and cerebral palsy. SCI can lead to temporary or permanent changes in spinal cord function, including numbness or paralysis. Currently, even with the best possible treatment, the injury generally results in some incurable impairment.

The research is co-led by Chew Sing Yian, principal investigator at SMART CAMP and associate professor of the School of Chemical and Biomedical Engineering and Lee Kong Chian School of Medicine at NTU, and Laurent David, professor at University of Lyon (France) and leader of the Polymers for Life Sciences group at CNRS Polymer Engineering Laboratory. The team includes CAMP principal investigators Ai Ye from Singapore University of Technology and Design; Jongyoon Han and Zhao Xuanhe, both professors at MIT; as well as Shi-Yan Ng and Jonathan Loh from Institute of Molecular and Cell Biology, A*STAR.

Chew says, Our earlier SMART and NTU scientific collaborations on progenitor cells in the central nervous system are now being extended to cell therapy translation. This helps us address SCI in a new way, and connect to the methods of quality analysis for cells developed in SMART CAMP.

Cell therapy, one of the fastest-growing areas of research, will provide patients with access to more options that will prevent and treat illnesses, some of which are currently incurable. Glaucoma and spinal cord injuries affect many. Our research will seek to plug current gaps and deliver valuable impact to cell therapy research and medical treatments for both conditions. With a good foundation to work on, we will be able to pave the way for future exciting research for further breakthroughs that will benefit the health-care industry and society, says Hanry Yu, co-lead principal investigator at SMART CAMP, professor of physiology with the Yong Loo Lin School of Medicine, NUS, and group leader of the Institute of Bioengineering and Nanotechnology at A*STAR.

The grants for both projects will commence on Oct. 1, with RAMP expected to run until Sept. 30, 2022, and ScaNCellS expected to run until Sept. 30, 2023.

SMART was. established by the MIT in partnership with the NRF in 2007. SMART is the first entity in the CREATE developed by NRF. SMART serves as an intellectual and innovation hub for research interactions between MIT and Singapore, undertaking cutting-edge research projects in areas of interest to both Singapore and MIT. SMART currently comprises an Innovation Centre and five interdisciplinary research groups (IRGs): Antimicrobial Resistance, CAMP, Disruptive and Sustainable Technologies for Agricultural Precision, Future Urban Mobility, and Low Energy Electronic Systems.

CAMP is a SMART IRG launched in June 2019. It focuses on better ways to produce living cells as medicine, or cellular therapies, to provide more patients access to promising and approved therapies. The investigators at CAMP address two key bottlenecks facing the production of a range of potential cell therapies: critical quality attributes (CQA) and process analytic technologies (PAT). Leveraging deep collaborations within Singapore and MIT in the United States, CAMP invents and demonstrates CQA/PAT capabilities from stem to immune cells. Its work addresses ailments ranging from cancer to tissue degeneration, targeting adherent and suspended cells, with and without genetic engineering.

CAMP is the R&D core of a comprehensive national effort on cell therapy manufacturing in Singapore.

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SMART researchers receive Intra-CREATE grant for personalized medicine and cell therapy - MIT News

Abu Dhabi Stem Cells Centre collaborating with Israeli firm for COVID-19 therapies – Gulf News

A medical staff member carries a swab sample for testing at a screening centre in Abu Dhabi.

Abu Dhabi: UAE's Abu Dhabi Stem Cells Centre (ADSCC) is collaborating with Israeli regenerative medicine company, Pluristem, to advance COVID-19 therapies.

In a statement, ADSCC said it was working to administer Pluristem developed PLX cells via a nebuliser to COVID-19 patients. The collaboration will allow ADSCC to expand its stem cell therapy options with the novel PLX cells, while also enabling Pluristem to leverage the ADSCCs nebuliser administration experience to develop a new treatment delivery model for PLX cells.

ADSCC has reported effective usage of nebulisers to treat patients suffering from COVID-19 infection, with stem cells sourced from the patients own blood.

Further discussions are also ongoing to treat chronic Graft Versus Host Disease (cGvHD) a life-threatening immune response to the donors stem cells against the host (patient).

Both projects follow a recently-signed agreement between ADSCC and Pluristem to harness the power of regenerative medicine.

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Abu Dhabi Stem Cells Centre collaborating with Israeli firm for COVID-19 therapies - Gulf News

Majority of Respondents Support Chimeric Animal Research: Survey – The Scientist

Human-animal chimeric embryosorganisms created using cells from two or more specieshave the potential to change how researchers study disease and generate organs and tissues for human transplants. One day, scientists have proposed, it may be possible for someone with, say, pancreatic cancer to have their stem cells injected into a modified swine embryo lacking its own pancreas so it can grow the human organ for donation.

Already, human-animal chimeric embryos (HACEs) have been created using human cells injected into pigs, sheep, mice, rats, and monkeys, although none in the US have been brought to term. In fact, their very existence is ethically contentious. What happens, for example, if scientists were to grow a human brain in an animal, blurring the line between species?

In response to ethical, social, and legal concerns, the National Institutes of Health (NIH) issued a moratorium on funding for HACE research in 2015 pending the development of a new set of regulatory guidelines. While research continues in other countriesand even in the US, through collaborations with foreign researchers and private fundingthe NIH has yet to reverse its decision, despite previous announcements that it would do so.

To gauge the American publics support for HACE research, Francis Shen, a professor of law at the University of Minnesota, recreated two recent surveys carried out in Japan, where many of the worlds HACE studies are done. In those surveys, Shens colleagues found that the majority of the Japanese public supported the use of HACEs, although their feelings varied depending on the type of organ or tissue being grown. We thought, Boy, itd be really interesting to see if the American public thinks about things the same way, Shen tells The Scientist.

Shens team directly translated the Japanese surveys into English, asking 430 participants to rate their support for each of the three steps involved in producing an organ using HACE technology: the insertion of human stem cells into an animal embryo, the transplanting of the embryo into a surrogate, and the harvesting of the resulting organ for use in a human. As before, they gauged peoples reactions to organ and tissue types, including skin, liver, blood, heart, brain, and gametes.

The Scientistspoke with Shen about the results of the US study, published today (October 1) in Stem Cell Reports.

University of Minnesota Law School

Francis Shen:Organ transplantation is a major goal, and that would be a major breakthrough. When I describe [HACEs] to . . . colleagues who maybe havent heard of them before, I talk about organ transplantation. And they understand that, yes, if you grow an organ from your own cells, it makes intuitive sense that your body might be more receptive.

I think there are also a large number of applications that fall broadly under regenerative medicine. One is to better understand the mechanisms of disease and organ function. Theres basic science advances to be achieved there. And then there are applied clinical advances and improvements in treatment across a wide range of diseases and disorders. We can develop better interventions, pharmacological and otherwise. The techniques are not just about improving organ donation. There are also a number of ways, through both basic and applied science, this work can really improve our knowledge and therefore response to any number of diseases and disorders.

FS: One of the main findings was that there seems to be very broad support, even broader than in the Japanese public, for the different steps of HACE research. Support was 59 percent, so a strong majority, support all three steps, including the returning of the organ into a human.

Second was that there are some differences across subgroups in the public. One thing that we thought was interesting is that, although lower, in some instances the support of those who are politically conservative was still quite strong . . . suggesting that this type of stem cell researchusing [induced pluripotent stem] cells and not embryonic stem cells harvested from a fetusperhaps could be more politically palatable.

FS:[People are] more accepting of using HACEs to grow things like livers and skin than sperm, eggs, or brains, and I think it gets to humanization. Its speculation, but I think its informed speculation based on other ethical scholarship and work that has been done. A liver is kind of a liver, it doesnt seem to have too many special properties, but the sperm and the egg and the brain, those are part and parcel of a person. So it feels much more ethically concerning to grow a person in a pig, as opposed to growing just some constituent part of a person.

Someone has a heart transplant or a liver transplant, and it just kind of seems to make more sense. I think theres a cultural acceptance and understanding thats easier to analogize to.

There are a series of ethical concerns, and we tried pretty hard in the paper to emphasize that. They include animal welfare, human dignity, and then the potential for the neurological humanization of chimeric animals.

I think addressing them is tremendously important. Although the NIH has a moratorium presently, it doesnt mean this type of research isnt going to happen. If the NIH doesnt fund it with these ethical guidelines in place, it could be funded and produced elsewhere outside of NIH purview. So not only is there an opportunity to set the ethics frameworks here, theres almost an imperative to do so.

The bottom line for me is that the ethics and the laws and regulation should go hand in hand with the development of the research. And to do that productively means we need to get together and work through these complicated issues. What our study suggests is that the American public would like us to do that.

Editors note: The interview was edited for brevity.

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Majority of Respondents Support Chimeric Animal Research: Survey - The Scientist

Dust off the crystal ball: It’s time for STAT’s 2020 Nobel Prize predictions – STAT

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

Senior Writer, Science and Discovery

Sharon covers science and discovery.

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Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Corporate Upd – PharmiWeb.com

NEW YORK, Oct. 2, 2020 /PRNewswire/ --BrainStorm-Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company will hold a conference call to update shareholders on financial results for the third quarter ended September 30, 2020, and provide a corporate update, at 8:00 a.m., Eastern Daylight Time (EDT), on October 15, 2020.

BrainStorm's CEO, Chaim Lebovits, will present a corporate update, after which, participant questions will be answered. Joining Mr. Lebovits to answer investment community questions will be Ralph Kern, MD, MHSc, President and Chief Medical Officer, David Setboun, PharmD, MBA, Executive Vice President and Chief Operating Officer, and Preetam Shah, PhD, MBA, Executive Vice President and Chief Financial Officer.

Participants are encouraged to submit their questions prior to the call by sending them to:q@brainstorm-cell.com. Questions should be submitted by5:00 p.m. EDT,Tuesday, October 13, 2020.

Teleconference Details BRAINSTORM CELL THERAPEUTICS 3Q 2020

The investment community may participate in the conference call by dialing the following numbers:

Participant Numbers:

Toll Free: 877-407-9205 International: 201-689-8054

Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website atwww.ir.brainstorm-cell.comand clicking on the conference call link.

Event Link: Webcast URL: https://bit.ly/30pVpNG Webcast Replay Expiration: Friday, October 15, 2021

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

Replay Number:

Toll Free: 877-481-4010 International: 919-882-2331 Replay Passcode: 37811

Teleconference Replay Expiration:

Thursday, October 29, 2020

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from theU.S. Food and Drug Administration(FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and initiated enrollment inMarch 2019.

AboutBrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(FDA) and theEuropean Medicines Agency(EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019. For more information, visit the company's website atwww.brainstorm-cell.com.

ContactsInvestor Relations: Corey Davis, Ph.D. LifeSci Advisors, LLC Phone: +1 646-465-1138 cdavis@lifesciadvisors.com

Media:Paul Tyahla SmithSolve Phone: + 1.973.713.3768 Paul.tyahla@smithsolve.com

View original content:http://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-to-announce-third-quarter-financial-results-and-provide-a-corporate-update-301144524.html

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm Cell Therapeutics to Announce Third Quarter Financial Results and Provide a Corporate Upd - PharmiWeb.com

Different Types of Childhood Cancer | INTEGRIS – Integris

If youre a parent, you know the thoughts, feelings and emotions that come with protecting your child. Youd do anything for them to ensure they live a long, healthy life. But what will you do when there are times when health conditions, such as childhood cancer, stand in the way?

Cancer in children is more common than you may think, as it accounts for the second-leading cause of death behind accidents. While a cancer diagnosis, or even the thought of your child developing cancer one day, is overwhelming, modern medicine has improved survival rates. As with any cancer, early detection is key. To help you familiarize yourself with childhood cancer, we asked Michael Confer, M.D., a radiation oncologist at the INTEGRIS Cancer Institute, about the different types of cancers, which signs to look for and how they can be treated.

Unlike adult cancers, which can result from the environment or exposure to certain things, childhood cancers result from genetic mutations that occur early on in life or before birth.

It all comes down to changes in genes. Your DNA contains information to make different types of cells in your body. In other words, your skin cells contain information to be brain cells, while your kidney cells contain information to be heart muscle cells. As cells mature, they become specialized, turning on and off certain genes to allow them to perform specific duties. Cells need to be able to replicate to replace damaged cells of the same category. They grow with help from genes called proto-oncogenes.

When your DNA changes, it leads to genetic mutations, and cells can become permanently activated. This can lead to cells duplicating uncontrollably, known as cancer.

Tumor suppressor genes slow down cell division. They repair DNA mistakes before cells divide and control cells internal death process (apoptosis or programmed cell death), Dr. Confer says. DNA mutations within tumor suppressor genes can also allow cells to duplicate uncontrollably. Children can be born with mutated proto-oncogenes or tumor suppressor genes in certain cells. These abnormally programmed cells lead to most childhood cancers.

So, what causes DNA changes? Your child can inherit genes from a parent that increases their risk of cancer or they can acquire these genes. Cancers from acquired, sporadic gene mutations are more common than those from inherited gene mutations 5% of all childhood cancers come from inherited mutations.

Breast cancer and ovarian cancer are the most common types of cancer caused by inherited DNA changes from BRCA1 or BRCA2 gene mutations. Even with how well-known these are, only 5 to 10% of breast cancer cases come from BRCA1/BRCA2 inherited mutations. Plus, breast cancer and ovarian cancer are more common in adults than children. Talk to your doctor or visit a genetic counselor if you have specific questions about inherited mutations.

Cancer can impact any part of your body, ranging from your bones and blood cells to your brain, spinal cord and other internal organs. You may be most familiar with leukemia, lymphoma, and brain and spinal tumors, since they are the most common. But, here is a full overview of cancers that commonly affect children, according to the American Cancer Society.

Leukemia: This is the most common type of cancer in children, accounting for 28% of cases. It generally starts in white blood cells and becomes fast growing (acute). Acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML) are the two most common types of leukemia. Three out of every four children with leukemia have ALL. This type of cancer starts in the lymphoid cells, called lymphocytes, whereas AML starts in myeloid cells. Chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL) are two types of rare cancers

Brain and spinal cord tumors: These types of cancers make up 26% of all cases. Brain tumors are more common than spinal cord tumors. The cancer generally starts in the lower part of the brain.

Neuroblastoma: This type of cancer affects infants and young children. Neuroblastoma starts in nerve cells during pregnancy and accounts for 6% of childhood cancers. Abdomen swelling is a common sign of neuroblastoma.

Wilms tumor: This type of cancer starts in the kidneys and impacts children who are 3 or 4 years old. It accounts for 5% of childhood cancers.

Lymphoma: Although lymphoma isnt as common as other types of cancers, youve likely heard of Hodgkin lymphoma and non-Hodgkin lymphoma, the two main types of cancer that show up in the lymphocytes. Non-Hodgkin lymphoma (accounts for 5% of childhood cancers) appears in younger children and is more common than Hodgkin lymphoma (accounts for 3%), which is more common in younger adults. You may notice a swollen lymph node under your childs arm or near their throat.

Rhabdomyosarcoma: This type of cancer develops in areas that your child uses to move their body, such as the head, pelvis, arms or legs. It accounts for 3% of childhood cancer cases.

Retinoblastoma: This cancer develops in the eyes, and your child is most at risk around the age of 2 until the age of 6. It accounts for 2% of childhood cancers.

Bone cancer: Bone cancer is more prominent in teens, but it still accounts for 3% of childhood cancers. There are two types, osteosarcoma and Ewing sarcoma, that show up via swelling around the bones. Ewing sarcoma is a less common type of cancer that is more prominent in older children and younger teens. Osteosarcoma, meanwhile, is more common in teens and in areas where bones grow quickly.

There isn't a one-size-fits-all guide to know if your child has cancer. In general, Dr. Confer says to keep an eye on any changes in your childs behavior, such as walking, eating, playing or sleeping. If theyre older, listen to any complaints they may have. Some cancers may produce a lump or swelling and pain in certain areas. Other symptoms include a loss of energy, weight loss, sudden eye or vision changes, frequent headaches with vomiting or a persistent fever that signifies the body is fighting an infection.

For example, leukemia, the most common type of childhood cancer, affects most children between the ages of 2 and 4. Typical symptoms include fever, bleeding, deep pain in the bones, small red spots on the skin called petechiae, bruises and enlarged lymph nodes.

Notify your childs pediatrician if any of these concerns arise. Aside from that, you should schedule your child for routine checkups and wellness visits.

Routine checkups and wellness visits help monitor normal growth and development. A good pediatrician-patient relationship helps the physician better identify subtle signs of cancer and gives parents a trusted sounding board for the concerns parents or children may have, Dr. Confer says.

Many childhood cancers have become increasingly treatable, leading to longer survival rates. Dr. Confer says acute lymphoblastic leukemia, lymphoma or kidney tumors known as Wilms tumor all have more than a 90% five-year survival rate (the percentage of patients who are alive five years after receiving treatment or a diagnosis).

In fact, the overall five-year survival rate for childhood cancers has improved from 58% in the mid-1970s to 84%, according to the American Cancer Society. But, certain types of aggressive cancer still exist. Diffuse intrinsic pontine glioma (DIPG), a rare brain tumor, is often cited as the childhood cancer with the poorest survival rate (less than 1% for five years).

No matter the diagnosis, continual hope and quality, proven therapies are the most important factors for children and families facing childhood cancers, Dr. Confer says.

Here are some of the most common forms of therapies to treat childhood cancer.

Surgery can help many patients, whether you need an entire tumor removed or a procedure to ease pain caused by a tumor. Your childs surgeon can also debulk a tumor, meaning they remove part of it and treat the rest with another method. Surgery has the highest success rate when its contained to one area, before the cancer has an opportunity to metastasize (spread to other parts of the body).

High doses of radiation help reduce cancer by either killing the cells or damaging their DNA to slow growth. Over time, these cells die and your body removes them. You can either receive internal or external radiation. External radiation comes from a beam that treats a specific body part, whereas internal radiation is in solid or liquid form. More specifically, brachytherapy is the medical term for solid internal radiation. Your doctor will place capsules, seeds or ribbons near the tumor. Systemic therapy is the medical term for liquid internal radiation. With this method, the radiation travels through your blood via a pill, injection or IV to kill cancer cells.

Chemotherapy comes in many methods of application, such as IV, oral, injection, topical or through a catheter, port or pump. Chemotherapy also kills healthy cells, which is one of the downsides. This is why many chemotherapy patients lose their hair and experience other side effects. Depending on the type and progression of the cancer, chemotherapy can help shrink a tumor to increase the success rate of surgery or radiation. Chemotherapy can also fight against any lingering cancer cells following surgery or radiation. Its also used to treat cancer that returns or metastasizes.

The immune system is your bodys way of defending itself against harmful germs, bacteria and viruses. When it comes to cancer, the immune system can have trouble recognizing and fighting off harmful cells because cancer starts in healthy cells. Immunotherapy helps your body pinpoint cancer kills to better defend against them. There are many types of immunotherapy treatments to boost your immune system. One type, chimeric antigen receptor (CAR) T-cell therapy, mixes your own T-cells with a virus that teaches the T-cells how to kill cancer cells.

Targeted therapy is a form of chemotherapy. But, as the name suggests, these drugs zero in on a specific area of the cancer cells. Depending on the drug, targeted therapy can change the protein levels in cancer cells or block chemical signals that help cancer cells grow. Other targeted therapy drugs can limit blood vessel production to cut off the cancer cells or distribute toxins to specifically kill the cancer while sparing healthy cells.

Stem cells, which originate in the bone marrow, make red blood cells, white blood cells and platelets. Leukemia and lymphoma start in the blood cells, causing damage to the cells your body needs to function. A stem cell transplant involves destroying cancer cells via chemotherapy and/or radiation before replacing them with new, healthy cells. This allows doctors to use stronger doses of chemotherapy or radiation knowing new cells, via a transplant, will replace old, damaged cells. Stem cell transplants can come from your own cells or the cells of another person. Donated cells are often more effective since they can help kill off cancer cells.

While you cant do anything to prevent your child from developing cancer, you can be proactive by scheduling regular checkups and looking out for warning signs and symptoms. Contact an INTEGRIS pediatrician if you have any concerns, and they can refer you to an oncologist at the INTEGRIS Cancer Institute.

Subscribe for regular emails full of useful and interesting Oklahoma-centric health and wellness info, from the doctors and health experts at INTEGRIS.

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Different Types of Childhood Cancer | INTEGRIS - Integris